This news can’t be overstated. Of course, one needs to question what the words “significant” means. Because it can be different things to different people.
Significant: “sufficiently great or important to be worthy of attention; noteworthy”.
In the pharma community significant means sometimes p<=0.05 even though this number is not written in stone the community has crowned it as the whole grail. Of course, we can refer to this for one patient (it worked for this person), two patients (it works for two patients), or, in the case of many people IT WORKS.
So, why is this great news?
To start with: it works in people !!!. Animal models in cancer clinical trials are the trickiest of all as safety and efficacy identified in animal studies is generally not translated to human trials. We knew that the murine xenograft experiment published by CYDY showed an efficacy of 98% in reducing metastatic tumor volume (explaining why we got fast track designation). Well, great however …. the average rate of successful translation from animal models to clinical cancer trials is less than 8%.
“The major pre-clinical tools for new-agent screening prior to clinical testing are experimental tumors grown in rodents. Although mice are most commonly used, they are actually poor models for the majority of human diseases. Crucial genetic, molecular, immunologic and cellular differences between humans and mice prevent animal models from serving as effective means to seek for a cancer cure. Among 4,000+ genes in humans and mice, researchers found that transcription factor binding sites differed between the species in 41% to 89% of cases. In many cases, mouse models serve to replicate specific processes or sets of processes within a disease but not the whole spectrum of physiological changes that occur in humans in the disease setting”
Second: it’s safe (some of you will say that this is not big news … well, LOTS of trials die simply because the drug is too toxic, we have grown used to the fact that Lero can be taken without SAEs but this on its own is a BIG deal (that is why Lero is potentially a blockbuster).
Let’s hope CDY releases the CTC progression for the patient(s) in due time. Let’s hope this number is going low (<5), and, down the road, when we have more patients, that p<0.05. This will give BP something to work on deciding, among other things, how humble do they have to come to talk to us …and, as NP says: how arrogant do we have to feel …